In 2016, Sarepta Therapeutics was given the green light by the FDA for its Duchenne muscular dystrophy (DMD) drug, Exondys 51. It was a huge win for the DMD patient community, as it was the first drug ever to receive FDA approval to treat the disease. However, Exondys 51 only helps 13% of DMD patients, meaning that the majority of the patients were left without an effective treatment option; until now.
Marathon Pharmaceuticals has recently announced that it has received FDA approval for its corticosteroid, Emflaza, known generically as deflazacort, which has been approved to treat all DMD patients in the US aged 5 and older.
This should be great news for the DMD patient community. However, as has been the case with so many of the drugs approved to treat rare diseases in the past year or so, pricing is getting in the way of patient access.
Deflazacort is not a new drug, and has been available to patients elsewhere in the world for many years. Even within the US, DMD patients have been accessing the drug, prior to its FDA approval, by importing it from abroad for around $1,200 per year. Marathon also had an Expanded Access Program in place, which was providing the drug for free to around 800 US patients.
Due to FDA regulations, however, patients will no longer be able to import the drug, or receive it through an Expanded Access Program, now that Marathon has marketing approval for the US. As such, patients will be forced to pay Marathon’s whopping list price of $89,000 per-year for the drug. This has caused major public backlash, and has raised some serious questions about Marathons pricing strategy.
To add to this, critics have also argued that the drug was wrongly awarded orphan drug status, which will give the drug a 7-year market exclusivity period, during which cheaper alternative drugs will be kept off the market. Orphan drug status is supposed to be reserved for new drug developments, and, since, deflazacort has been available for years, the argument has been made that it is not, in fact, a new drug.
It is easy to see why patients might be distressed, given that drug prices seem to be rising astronomically year on year. However, Marathon has justified the high price of its drug by claiming that substantial resources were invested into getting the drug approved in the US, and it does not expect to recoup this investment for several years. It also made efforts to appease the growing concerns by (a) promising to work with payers and patients to make Emflaza accessible to all families, regardless of their ability to pay, and (b) pledging to reinvest any profit made into more research in the DMD space.
It seems Marathon has had little success convincing industry and the patient community, however, as earlier this week, the company announced that it will pause the launch of its drug. In his statement, Marathon CEO, Jeff Aronin, reassured patients that the company is delaying the launch in order to now meet with caregivers and advocacy groups to review their concerns, discuss all options, and create an appropriate plan of action for moving forward with commercialization.